The tumor site-agnostic clinical development of TRK inhibitors for pediatric solid tumors is a current example of how the combination of genetic testing and innovative clinical trial design can accelerate the clinical development of targeted agents for pediatric patients.
Preliminary findings from a phase I study of LOXO-195 show that the investigational agent appears effective in patients with <i>NTRK</i> fusion-positive solid tumors that have become resistant to first-generation TRK inhibitors.
The FDA has approved entrectinib for patients with any solid tumor bearing <i>NTRK</i> fusions, making it the second TRK inhibitor to receive a tissue-agnostic approval; larotrectinib was the first.
Although TRK fusions occur in fewer than 1% of all solid tumors, inhibition of TRK results in profound therapeutic responses, resulting in Breakthrough Therapy FDA approval of the TRK inhibitor larotrectinib for adult and pediatric patients with solid tumors, regardless of histology.